When Congress passed the Right to Try Act during the first Trump administration, the goal was straightforward: give patients with serious or rare diseases a fair chance to access promising therapies when conventional options had been exhausted. The policy reflected a broader philosophy that speed, flexibility, and compassion should play a larger role in drug regulation — especially where the alternative for patients was simply waiting for a progressive decline.
The philosophy of President Trump has not meaningfully changed. But the way some officials within the Food and Drug Administration (FDA) have been acting during his second administration increasingly suggests a different mindset. In recent years, agency leaders such as Vinay Prasad, who serves as Chief Medical and Scientific Officer at the FDA and Director of the Center for Biologics Evaluation and Research, have expressed skepticism about accelerated approvals and have argued that the FDA has historically been too permissive. A healthy scientific debate about standards is appropriate – but problems arise when that skepticism hardens into regulatory caution that slows access even where evidence points toward meaningful benefits.
Drug development for rare diseases is inherently arduous. Trials are small. Patient populations vary widely. Real-world disruptions — including the COVID pandemic — have complicated data collection and sometimes weakened statistical signals even when biological effects remain clear. Regulators have traditionally acknowledged this reality by weighing the totality of evidence rather than expecting the same scale and statistical certainty typical of trials for more common diseases. Lately, however, that balance appears to be shifting.
The ongoing controversy surrounding Sarepta Therapeutics’ gene therapy Elevidys illustrates the dilemma. Duchenne muscular dystrophy (DMD) is a devastating genetic disease that progressively robs children of muscle function, typically leading to the loss of mobility in adolescence with serious cardiac and respiratory complications to follow. Families facing that diagnosis are not debating abstract regulatory theory; they are searching for time, stability, and hope.
Elevidys was developed to address the root genetic cause of DMD by enabling production of a functional microdystrophin protein. Early data showed encouraging biological effects and functional signals that suggested slower disease progression. In 2025, however, the FDA halted distribution after safety concerns emerged in a small subset of patients, prompting the agency to reassess whether the therapy’s benefits continued to outweigh its risks.
Safety oversight is essential, and serious adverse events deserve careful scrutiny. In the Elevidys case, however, the fatalities that triggered concern occurred primarily among patients with more advanced disease and other underlying vulnerabilities that can complicate gene therapy treatment. Sarepta had already responded to these fatalities by voluntarily pausing use in the most at-risk older patients prior to the FDA’s order, yet the agency proceeded with a broader halt even though no new safety issues had emerged among younger ambulatory patients most likely to benefit from the therapy. Rather than incorporating that nuance, FDA’s sweeping action basically treated class-related gene therapy risks as disqualifying, leaving patients suffering from this debilitating disease with few effective treatment options.
Now, even as recently released EMBARK trial data provides additional findings that indicate sustained benefits and manageable safety risks, the FDA has been slow to reconsider its position. That disconnect fuels concern not only about this therapy but about regulatory consistency at the agency more broadly.
Accelerated approvals for treatments in oncology and rare disease settings have a long history at the FDA when the unmet medical need was acute. Conversely, isolated adverse events have historically been contextualized rather than treated as definitive. When a therapy appears unable to move forward, even after additional positive data, it creates the impression that the standards themselves may be shifting.
That perception matters, not just for patients but also for innovation. Biotech development is expensive, risky, and often driven by investor confidence that regulatory pathways are predictable. If sponsors begin to believe approvals hinge as much on philosophical swings as on scientific evidence, investment flows elsewhere. And when innovation migrates and disperses, patients lose first.
The solution is not to abandon rigorous standards. Drug safety must remain paramount. But Right to Try was never about bypassing science; it was about recognizing that patients facing life-limiting diseases often accept risk differently than regulators do. A system that ignores that reality risks substituting bureaucratic preference for patient judgment. Restoring balance will require a shift in both tone and action.
The FDA should revisit decisions like the Elevidys pause in light of updated trial data and do so transparently. This would help restore confidence and demonstrate that regulatory decisions remain responsive to credible scientific evidence. The agency should reaffirm a consistent, accelerated approval philosophy that aligns with the original Right to Try ethos: clear evidentiary expectations, predictable review standards, and meaningful engagement with patients whose lives are directly affected by these decisions.
America’s leadership in biomedical innovation has always depended on two things: strong science and a willingness to move decisively when patients cannot afford delay. Losing either undermines both and underscores why Right to Try was never simply a legislative milestone, but a statement about how we value patients confronting devastating illnesses. Regulators should keep that principal front and center remembering that their role is to serve patients, not stand in their way.
Becky Corbin previously served in the Pennsylvania House of Representatives where she was a member of the Health Committee and the Rare Disease Caucus. A chemist by training, she previously worked in the pharmaceutical industry.
